Balancing the risks and benefits of prescription drugs

The depiction of Judgment Day on the façade of the Cathedral of Notre Dame in Paris shows an angel weighing the good and evil of souls to determine where they should go. Hanging on one side are devils tilting the scale in the wrong direction.

In making decisions about treating neurologic illnesses, physicians and their patients weigh risks and benefits of any treatment. For us, the devil is in the details of those risks and benefits. If we underestimate risks or overestimate benefits, the scales can tilt in the wrong direction.

How do physicians make decisions about benefits and risks? For drugs that are
approved by the Food and Drug Administration (FDA) for a given condition, we have good information about the benefit of the given drug based on the research done to get FDA approval. This research typically examined the benefits of the drug in comparison to a placebo and involved hundreds of patients who received the drug or placebo.

This assessment of benefit, however, is not perfect. The clinical research gives information about the benefits of the drug to a group of patients, whereas physicians have to decide the probability that the drug will help one patient — namely the person sitting in front of us waiting for our recommendation. That person may be quite different from the people who were enrolled in the clinical trial.

The people enrolled in clinical trials have to meet specific enrollment criteria and our specific patient may be quite different. He or she may be older, may have other illnesses or be taking other medications that might decrease the benefit of the drug.

Physicians also commonly use medications that are FDA approved for one illness to treat another condition, referred to as “off label” use. For instance, many anti-seizure medications are used to treat certain types of pain. Sometimes there is good evidence from research that this “off-label” use is beneficial. Other times the physician is basing the decision on personal experience and limited published research. “Off-label” use of medications, while common, is often based on less extensive published experience, making decisions about benefit challenging.

What about risks? How do physicians make decisions about risks of therapy? This is even more challenging than predicting benefit, particularly for new medications.

Information about side effects from medications is gathered during clinical trials of the drug. But this information is just the beginning. Because the initial clinical research may have involved only a few hundred patients, it is not until the drug is FDA approved and used in many more patients that rarer and serious side effects may become apparent. The drug may also be used in older or sicker patients than those in the clinical trials and these individuals may be more likely to experience side effects. It is only with time that we gain a full appreciation of the risks of drugs.

So what is a patient to do? When discussing a treatment with your physician, find out whether or not the drug is FDA approved for the condition your doctor is prescribing it for. If not, find out whether there is any research supporting its use and what your doctor’s experience has been with the drug for this “off-label” use.

Find out how long the drug has been on the market. If it is a new drug, you should be aware that less is known about its safety than a drug that has been around for a few years or longer. Find out if there is an alternative drug that may have a longer safety track record and, if so, ask why your doctor is recommending the new drug. Finally, be clear on what benefits you should expect from the drug and also what the common side effects and potential serious side effects are for the drug.

There is an old adage for doctors: “Be neither first nor last to prescribe a new drug.” We avoid being first so there is time to learn about side effects. Being last means that we would not be keeping up and taking advantage of true treatment advances.  This adage can be modified for patients to: ”Don’t be the first to take a new drug unless there is a very good reason to do so, and don’t be the last if the new drug can help you.”

Dennis Bourdette, M.D.
Professor and Chair, Department of Neurology
OHSU Brain Institute